BioCentury | Oct 18, 2018
Distillery Techniques

Biomarkers; other

...spliced in DM1 patients, but not in healthy volunteers or other muscular dystrophy patients, including muscleblind-like splicing regulator 1 (MBNL1)...
...2018 doi:10.1038/s41467-018-06206-0 CONTACT: Thurman M. Wheeler, Massachusetts General Hospital, Boston, Mass. email: twheeler1@mgh.harvard.edu Claire Quang Massachusetts General Hospital Muscleblind-like splicing regulator 1 (MBNL1) Nuclear...
BioCentury | Mar 8, 2018
Emerging Company Profile

Splice time

...Inc − have productss that modulate SMN2 splicing and promote activity of the splicing-related protein MBNL1...
...Plainfield, N.J. Roche (SIX:ROG; OTCQX:RHHBY), Basel, Switzerland Skyhawk Therapeutics Inc., Waltham, Mass. Targets MBNL1 - Muscleblind-like splicing regulator 1...
BioCentury | Jan 3, 2018
Product R&D

Expanding small molecule horizons

...pre-mRNAs, resulting in low levels of MBNL1 in the nucleus. (4) The reduction in free MBNL1...
...DM1 pathology. CLCN1 - chloride voltage-gated channel 1; DMPK - dystrophia myotonica-protein kinase; MBNL1 - muscleblind-like splicing regulator 1...
...Finger Nucleic Acid Binding Protein DMPK - Dystrophia myotonica-protein kinase KRAS - K-Ras MBNL1 - Muscleblind-like splicing regulator 1...
BioCentury | Sep 14, 2017
Emerging Company Profile

RNA gets CRISPR

...which triplet CTG repeats in the DMPK gene create CUG repeat-containing RNA foci that sequester MBNL1...
...and Compounds Mentioned Cas9 - CRISPR-associated protein 9 DMPK - Dystrophia myotonica-protein kinase MBNL1 - Muscleblind-like splicing regulator 1...
...pathology by RNA-targeting Cas9 in a myotonic dystrophy mouse model.” bioRxiv (2017) Lauren Martz, Senior Writer Locana Inc. Muscleblind-like splicing regulator 1 (MBNL1)...
BioCentury | Jun 29, 2017
Translation in Brief

New Therapeutic Targets and Biomarkers: June 2017

New Therapeutic Targets and Biomarkers: June 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during June 2017. Therapeutic targets are defined as any protein, gene or other...
BioCentury | Jun 7, 2017
Distillery Therapeutics

Cancer

INDICATION: Liver cancer Patient sample, cell culture and mouse studies suggest inhibiting MBNL3 could help treat hepatocellular carcinoma (HCC) and other liver cancers. In tissue samples from HCC patients, levels of MBNL3 were higher in...
BioCentury | May 15, 2014
Distillery Therapeutics

Indication: Musculoskeletal disease

...by CUG repeats in the DMPK mRNA that bind and sequester the splicing regulator muscleblind (MBNL1...
...cells expressing CUG repeat-containing DMPK, an inhibitor that bound the repeats and blocked binding to MBNL1...
BioCentury | Oct 4, 2012
Distillery Techniques

Technology: Disease models

...DM1 could be useful for studying neurological features of the disease. Mice lacking muscleblind-like 1 (Mbnl1...
...Next steps could include testing the effect of candidate DM1 therapeutics on neurological function in Mbnl1...
BioCentury | Oct 15, 2009
Distillery Therapeutics

Indication: Musculoskeletal disease

...Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Muscleblind-like 1 (MBNL1...
...myotonica protein kinase (DMPK) Studies in cell culture and in mice suggest that inhibitors of MBNL1...
BioCentury | Sep 17, 2009
Distillery Therapeutics

Indication: Musculoskeletal disease

...Publication and contact information Musculoskeletal disease Muscular dystrophy Dystrophia myotonica protein kinase (DMPK); muscleblind-like 1 (MBNL1...
...dystrophy type 1 (DM1), a disease caused by aberrant interactions between DMPK mRNA and the MBNL1...
...protein. Based on X-ray crystal structures, a ligand was developed that destabilized the complex between MBNL1...
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